Health and Healthcare

What's Next for Sangamo After This Hemophilia Study

Chris Lange

Published: April 2, 2019 11:40 am

Last Updated: January 30, 2020 10:35 pm

Sangamo Therapeutics Inc. (NASDAQ: SGMO) shares jumped on Tuesday after the company announced interim data from its midstage hemophilia trial in collaboration with Pfizer Inc. (NYSE: PFE). The Phase 1/2 Alta study is evaluating investigational SB-525 gene therapy for severe hemophilia A.

Ultimately, the data indicate that SB-525 was generally well-tolerated and demonstrated a dose-dependent increase in Factor VIII levels across the four dosage cohorts. Eight patients total were dosed.

Patients in the Alta study were not treated with prophylactic steroids. No treatment-related serious adverse events were observed in the first three cohorts.

In addition to the partnership for the development and commercialization of gene therapies for hemophilia A, Sangamo and Pfizer are also collaborating on the development of gene therapies for amyotrophic lateral sclerosis (ALS) and frontotemporal lobar degeneration using Sangamo’s proprietary zinc finger protein transcription-factor technology.

Edward Conner, M.D., chief medical officer of Sangamo, commented:

The interim data from the first eight patients with hemophilia A treated with SB-525 gene therapy in the Alta study are encouraging and demonstrate a dose-dependent relationship, evidence of sustained factor levels, and low variability, both within each patient and within each cohort. These interim results suggest that SB-525 may be well-tolerated and may prove to have the predictability and sustained treatment effect that can bring clinical benefit in patients with hemophilia A. We need to continue observing how the data mature and how additional patients in the expansion cohort respond to SB-525. We look forward to working with Pfizer to potentially advance SB-525 into a registrational study.

Shares of Sangamo were last seen up about 29% at $12.28, in a 52-week range of $6.26 to $20.55. The stock has a consensus price target of $18.71.

Pfizer traded at $42.97 a share. The 52-week range is $34.37 to $46.47, and the consensus price target is $44.14.